FDA warp speed RMAT approval nukes stem cell clinic excuses

Many stem cell clinics are in a bind in 2017 in the new RMAT approval reality.

warp speed

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Stem cell clinics say that they only want to help people by providing stem cell therapies to patients in a speedier way that they claim getting FDA approval for a trial just doesn’t allow, but that doesn’t ring true. It never has and now even less so.

The new RMAT designation out there based on the 21st Century Cures Act whereby the FDA can rapidly accelerate truly promising stem cell therapies in clinical trials makes this stem cell clinic excuse of the need for speed seem even less based on common sense. For more on RMATs see this background post. With RMAT, one eligibility criterion for that designation is that the illness in question must be serious or life threatening. The clinics have generally indicated in the past that what they treat are serious conditions. Will the FDA see it that way? It’ll depend on the conditions in each case.

The FDA has already approved 4 RMATs in just a few months so they are not holding back. If you really have good, solid clinical data for a serious or life-threatening condition, then you should apply to the FDA for an RMAT and in theory you can have both speed and FDA approval for a trial to move quickly. This new situation makes that general stem cell clinic marketing pitch of needing to get supposedly truly promising stem cells to patients more quickly than the FDA system allows ring very hollow.

Clinical speed with no real data is kind of like driving a race car with no brakes.

And if you are a patient, before you consider getting any stem cell treatment you have a new additional “must-ask” question for the clinics out there: “Have you applied for an RMAT from the FDA?” or “Do you have RMAT approvals?” Also, see my stem cell treatment guide for patients where I list 10 more questions to ask and things to think about. Asking your primary care doctor who is not involved in the stem cell treatment for their professional take in advance on your consideration of a stem cell treatment is a good idea.

Clinics can’t retract stem cell treatments gone bad

Stem cell facelift comicYou can stop taking a pill or an injection treatment, but you can’t stop or retract stem cell treatments if there’s a bad side effect.

Unlike other kinds of medicines, once stem cells have been transplanted into patients, if something goes wrong you cannot stop the ‘treatment’. There’s no retraction possible because transplanted stem cells spread in the body and potentially integrate.

One of the striking things in the commercial stem cell arena in 2016 was the emergence of patient lawsuits against stem cell clinics including two proposed class action suits. These patients, and I count potentially now more than a dozen, allege a variety of harms ranging from tumors to blindness. The reason I mention this is that there appears to be huge potential for harm to patients from unapproved stem cell therapies. I know a lot of patients who would wish they could undo what the stem cell clinic did. It’s just not possible.Stem cell cartoon

Even in an appropriately regulated stem cell trial context, there’s no easy way to undo stem cell transplants. There has been talk for years about suicide genes to be inserted into stem cells to provide “a net” should something go awry with stem cell treatments, but it’s not clear how well these would work and stem cell clinics aren’t interested in that anyway.

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Stemgenex motion to dismiss aims to rebut stem cell suit claims

motion-to-dismiss-stemgenexSan Diego-based stem cell business Stemgenex is the subject of a suit over allegations about stem cell treatments. The company has now filed a motion to dismiss the case.

To me as a non-attorney, the Stemgenex motion to dismiss seems to emphasize overall vagueness of the plaintiff’s case and argues in part that there is insufficient detailed support of each claim.

From my reading of the plaintiff’s suit document, the case seems focused on alleged issues with Internet marketing claims and it provides details mainly on that level. Are all nine claims related to that? I’m not sure.

The motion to dismiss also asserts that there is a lack of detail on the specific alleged roles of each defendant as pertains to the nine claims. I’m more accustomed to reading, writing, and critiquing scientific materials (e.g. papers and grants), so court documents seem somewhat foreign to me and I don’t know what the expectations are for claims. I do feel like there are some lacunae in terms of specific details of the nine claims in the case, but is that par for the course at this stage?

Stay tuned as we all learn more about this situation and hopefully one or more experts in legal matters will weigh in on it.

South Africa grapples with “illegal” stem cell experiment scandal

dr-wian-standerSouth Africa is facing a stem cell scandal related to what a health policy news outlet called Spotlight there characterizes as illegal experiments by ReGenesis Biotechnologies, the company at the heart of the controversy. It had a contract with a governmental health provider agency, now apparently suspended.

The report begins this way: “The Medicines Control Council (MCC) this week suspended what appears to be unlawful stem cell experimentation at Pelonomi, a state hospital in Bloemfontein.”

However, there does not appear to be concrete data supporting safety and efficacy of the “treatments” in question. There were additional concerns over informed consent:

“MCC Chairperson Professor Helen Rees confirmed to Spotlight that inspectors had been to the Pelonomi site last Friday and again on Monday.

“Our concern was that the service level agreement made reference to medicines, injections and therapeutic research,” said Rees.

She said the informed consent documents referred to the patients giving permission for stem cell therapy, permission for stem cells to be removed, concentrated and re-injected and for their stem cells to be given to another person.”

The leader of the company was reported as Dr. Wian Stander.

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LA Times Shines Some Light on Stem Cell Clinics including StemGenex

A large and growing number of American clinics are selling stem cells to patients for a variety of ills and one in the San Diego area called StemGenex was the main focus of a recent LA Times piece by columnist Michael Hiltzik. In the piece called “These new stem cell treatments are expensive — and unproven” Hiltzik discussed the growing issues over stem cell clinics in the U.S. and he used StemGenex as a kind of test case or example.StemGenex

He started off with a description of the kind of hopeful feeling that many patients experience upon visiting stem cell clinic websites:

“Visitors to the website of StemGenex, a La Jolla medical group, could be forgiven for thinking that the answer to their prayers is finally at hand.”

However, there is little published data to support the expectation that one’s prayers might be answered at U.S. stem cell clinics today. I talked with Hiltzik about the state of the American stem cell clinic arena and my concerns as he was researching his piece. The marketing of stem cells is too aspirational in my view and patients are sold medical interventions in many cases that may not work and have potential risks. Hiltzik writes (emphasis mine):

“StemGenex’s director of media and community relations, Jamie Schubert, told me that its “principal purpose is helping people with unmet clinical needs achieve optimum health and better quality of life,” and that it has “anecdotal feedback … from our patients that their symptoms have dramatically improved and their quality of life has substantially increased.”

Keep in mind that we are talking about hundreds of clinics in the U.S. injecting living stem cells into patients’ bloodstreams or into specific tissues and most clinics out there do not have FDA approval to do this so anecdotes are not a strong foundation for this kind of practice in my opinion.

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